WO2000006705A3 - Genetisch modifizierte cd34-negative, adhärent wachsende stammzellen und deren verwendung in der gentherapie - Google Patents

Genetisch modifizierte cd34-negative, adhärent wachsende stammzellen und deren verwendung in der gentherapie Download PDF

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Publication number
WO2000006705A3
WO2000006705A3 PCT/DE1999/002309 DE9902309W WO0006705A3 WO 2000006705 A3 WO2000006705 A3 WO 2000006705A3 DE 9902309 W DE9902309 W DE 9902309W WO 0006705 A3 WO0006705 A3 WO 0006705A3
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Prior art keywords
stem cells
genetically modified
negative
gene therapy
therapy
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PCT/DE1999/002309
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English (en)
French (fr)
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WO2000006705A2 (de
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Ralf Huss
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Ralf Huss
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Application filed by Ralf Huss filed Critical Ralf Huss
Priority to JP2000562487A priority Critical patent/JP4012688B2/ja
Priority to US09/743,904 priority patent/US6361997B1/en
Priority to AU61872/99A priority patent/AU741168B2/en
Priority to CA002338541A priority patent/CA2338541C/en
Priority to EP99948706A priority patent/EP1100878B8/de
Priority to AT99948706T priority patent/ATE296875T1/de
Priority to DE59912118T priority patent/DE59912118D1/de
Publication of WO2000006705A2 publication Critical patent/WO2000006705A2/de
Publication of WO2000006705A3 publication Critical patent/WO2000006705A3/de

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/48Drugs for disorders of the endocrine system of the pancreatic hormones
    • A61P5/50Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20Cytokines; Chemokines
    • C12N2501/23Interleukins [IL]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Genetics & Genomics (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Communicable Diseases (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Die Erfindung betrifft die Verwendung von genetisch modifizierten, frühesten hämatopoetischen und mesenchymalen Stammzellen (negativ für die Expression des Oberflächenmoleküls CD34) in der individuellen Gentherapie von mono- bzw. oligogenetischen Erkrankungen oder in der Zelltherapie. Dazu werden aus dem peripheren Blut des Patienten körpereigene, CD34-negative, adhärent wachsende Stammzellkulturen angelegt und diese mit Genkonstrukten effizient transfiziert bzw. infiziert. Die Genprodukte dieser Gene sollen defekte oder fehlende Proteine bzw. Faktoren im Patientenorganismus langfristig substituieren. Die körpereigenen Stammzellen können nach Expansion auch zur Zelltherapie (Organersatztherapie) verwendet werden.
PCT/DE1999/002309 1998-07-24 1999-07-22 Genetisch modifizierte cd34-negative, adhärent wachsende stammzellen und deren verwendung in der gentherapie WO2000006705A2 (de)

Priority Applications (7)

Application Number Priority Date Filing Date Title
JP2000562487A JP4012688B2 (ja) 1998-07-24 1999-07-22 遺伝子修飾したcd34−陰性付着成長性幹細胞および遺伝子療法におけるそれらの使用
US09/743,904 US6361997B1 (en) 1998-07-24 1999-07-22 Genetically modified CD34-negative adherently growing stem cells and their use in gene therapy
AU61872/99A AU741168B2 (en) 1998-07-24 1999-07-22 Genetically modified CD34-negative adherently growing stem cells and their use in gene therapy
CA002338541A CA2338541C (en) 1998-07-24 1999-07-22 Genetically modified cd34-negative adherently growing stem cells and their use in gene therapy
EP99948706A EP1100878B8 (de) 1998-07-24 1999-07-22 Verfahren zur herstellung von genetisch modifizierten cd34-negativen, adhärent wachsenden hämatopoietischen stammzellen
AT99948706T ATE296875T1 (de) 1998-07-24 1999-07-22 Verfahren zur herstellung von genetisch modifizierten cd34-negativen, adhärent wachsenden hämatopoietischen stammzellen
DE59912118T DE59912118D1 (de) 1998-07-24 1999-07-22 Verfahren zur herstellung von genetisch modifizierten cd34-negativen, adhärent wachsenden hämatopoietischen stammzellen

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE19833476.1 1998-07-24
DE19833476A DE19833476B4 (de) 1998-07-24 1998-07-24 Genetisch modifizierte CD34-Negative, adhärent wachsende hämatopoetische Stammzellen und deren Verwendung in der Gentherapie

Publications (2)

Publication Number Publication Date
WO2000006705A2 WO2000006705A2 (de) 2000-02-10
WO2000006705A3 true WO2000006705A3 (de) 2000-05-04

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PCT/DE1999/002309 WO2000006705A2 (de) 1998-07-24 1999-07-22 Genetisch modifizierte cd34-negative, adhärent wachsende stammzellen und deren verwendung in der gentherapie

Country Status (9)

Country Link
US (1) US6361997B1 (de)
EP (1) EP1100878B8 (de)
JP (1) JP4012688B2 (de)
AT (1) ATE296875T1 (de)
AU (1) AU741168B2 (de)
CA (1) CA2338541C (de)
DE (2) DE19833476B4 (de)
ES (1) ES2242422T3 (de)
WO (1) WO2000006705A2 (de)

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WO2001005944A1 (en) * 1999-07-20 2001-01-25 University Of Southern California Identification of pluripotent pre-mesenchymal, pre-hematopoietic progenitor cell
US10638734B2 (en) 2004-01-05 2020-05-05 Abt Holding Company Multipotent adult stem cells, sources thereof, methods of obtaining and maintaining same, methods of differentiation thereof, methods of use thereof and cells derived thereof
US8252280B1 (en) 1999-08-05 2012-08-28 Regents Of The University Of Minnesota MAPC generation of muscle
US7015037B1 (en) 1999-08-05 2006-03-21 Regents Of The University Of Minnesota Multiponent adult stem cells and methods for isolation
US20020045260A1 (en) * 2000-10-17 2002-04-18 Shih-Chieh Hung Method of isolating mesenchymal stem cells
US7560280B2 (en) 2000-11-03 2009-07-14 Kourion Therapeutics Gmbh Human cord blood derived unrestricted somatic stem cells (USSC)
CA2438501C (en) * 2001-02-14 2014-09-16 Leo T. Furcht Multipotent adult stem cells, sources thereof, methods of obtaining and maintaining same, methods of differentiation thereof, methods of use thereof and cells derived thereof
US20070111201A1 (en) * 2001-04-30 2007-05-17 Benjamin Doranz Reverse transfection of cell arrays for structural and functional analyses of proteins
DE10144326B4 (de) * 2001-09-10 2005-09-22 Siemens Ag Verfahren und System zur Überwachung eines Reifenluftdrucks
ITRM20030376A1 (it) 2003-07-31 2005-02-01 Univ Roma Procedimento per l'isolamento e l'espansione di cellule staminali cardiache da biopsia.
US11660317B2 (en) 2004-11-08 2023-05-30 The Johns Hopkins University Compositions comprising cardiosphere-derived cells for use in cell therapy
US20100093089A1 (en) * 2006-11-09 2010-04-15 Eduardo Marban Dedifferentiation of adult mammalian cardiomyocytes into cardiac stem cells
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US9617506B2 (en) 2013-11-16 2017-04-11 Terumo Bct, Inc. Expanding cells in a bioreactor
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JP6878274B2 (ja) 2014-10-03 2021-05-26 シーダーズ−サイナイ・メディカル・センターCedars−Sinai Medical Center 筋ジストロフィーの処置における心筋球由来細胞およびこのような細胞によって分泌されたエキソソーム
WO2017004592A1 (en) 2015-07-02 2017-01-05 Terumo Bct, Inc. Cell growth with mechanical stimuli
EP3402543B1 (de) 2016-01-11 2021-09-08 Cedars-Sinai Medical Center Aus kardiosphäre stammende zellen und durch solche zellen sekretierte exosomen in der behandlung von herzversagen mit konservierter auswurffraktion
EP3464565A4 (de) 2016-05-25 2020-01-01 Terumo BCT, Inc. Zellexpansion
WO2017210652A1 (en) 2016-06-03 2017-12-07 Cedars-Sinai Medical Center Cdc-derived exosomes for treatment of ventricular tachyarrythmias
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Also Published As

Publication number Publication date
CA2338541C (en) 2007-04-03
AU741168B2 (en) 2001-11-22
EP1100878B1 (de) 2005-06-01
ATE296875T1 (de) 2005-06-15
AU6187299A (en) 2000-02-21
ES2242422T3 (es) 2005-11-01
DE19833476B4 (de) 2005-08-25
CA2338541A1 (en) 2000-02-10
US6361997B1 (en) 2002-03-26
DE59912118D1 (de) 2005-07-07
EP1100878A2 (de) 2001-05-23
DE19833476A1 (de) 2000-01-27
JP2003527817A (ja) 2003-09-24
JP4012688B2 (ja) 2007-11-21
EP1100878B8 (de) 2005-08-17
WO2000006705A2 (de) 2000-02-10

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